A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients (STAGED-PKD)
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ClinicalTrials.gov Identifier: NCT03523728 |
Recruitment Status :
Terminated
(Interim analysis for futility of the Stage 1 of the EFC15392 study met the protocol specified stopping rule based on the primary endpoint. EFC15392 study was stopped for futility based on prespecified criteria and recommendation from DMC)
First Posted : May 14, 2018
Results First Posted : November 9, 2022
Last Update Posted : November 9, 2022
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Sponsor:
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
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Study Type | Interventional |
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Study Design | Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Investigator, Outcomes Assessor); Primary Purpose: Treatment |
Condition |
Polycystic Kidney, Autosomal Dominant |
Interventions |
Drug: Venglustat Drug: Placebo |
Enrollment | 478 |
Participant Flow
Recruitment Details | This study was conducted at 93 sites that enrolled participants in 23 countries. A total of 478 participants were enrolled from 04 October 2018 to 01 June 2021. Study was conducted in 2 stages: Stage 1 and Stage 2. |
Pre-assignment Details | Participants who completed 24 months of treatment in either Stage 1 or Stage 2 of EFC15392 had option to enroll in an open-label long-term extension study LTS15823 (NCT ID: NCT04705051). |
Arm/Group Title | Stage 1- Placebo | Stage 1- Venglustat 8 mg | Stage 1- Venglustat 15 mg | Stage 2- Placebo | Stage 2- Venglustat 15 mg |
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Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months. | Participants from Stage 1 were randomized to receive venglustat 8 milligrams (mg) (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months. | Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months. | Participants from Stage 2 were randomized to receive 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months. | Participants from Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. |
Period Title: Stage 1 (24 Months) | |||||
Started | 78 | 78 | 80 | 0 | 0 |
Completed | 12 | 10 | 12 | 0 | 0 |
Not Completed | 66 | 68 | 68 | 0 | 0 |
Reason Not Completed | |||||
Adverse Event | 2 | 2 | 4 | 0 | 0 |
Progressive disease | 0 | 0 | 1 | 0 | 0 |
Lack of Efficacy | 2 | 0 | 0 | 0 | 0 |
Poor compliance to protocol | 1 | 0 | 0 | 0 | 0 |
Withdrawal by Subject | 6 | 5 | 7 | 0 | 0 |
Study terminated by sponsor | 54 | 59 | 56 | 0 | 0 |
Other-Unspecified | 1 | 2 | 0 | 0 | 0 |
Period Title: Stage 2 (24 Months) | |||||
Started | 0 | 0 | 0 | 123 [1] | 119 [1] |
Treated | 0 | 0 | 0 | 122 | 119 |
Estimated Glomerular Filtration Rate (eGFR) Between 45 and 89.9 mL/Min/1.73 m^2 at Screening | 0 | 0 | 0 | 97 | 90 |
eGFR Between 30 and 44.9 mL/Min/1.73 m^2 at Screening | 0 | 0 | 0 | 25 | 29 |
Completed | 0 | 0 | 0 | 0 | 0 |
Not Completed | 0 | 0 | 0 | 123 | 119 |
Reason Not Completed | |||||
Adverse Event | 0 | 0 | 0 | 1 | 2 |
Withdrawal by Subject | 0 | 0 | 0 | 0 | 4 |
Study terminated by sponsor | 0 | 0 | 0 | 119 | 110 |
Other-Unspecified | 0 | 0 | 0 | 2 | 3 |
Randomized and not treated | 0 | 0 | 0 | 1 | 0 |
[1]
Stage 1 and Stage 2 were separate population.
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Baseline Characteristics
Arm/Group Title | Stage 1- Placebo | Stage 1- Venglustat 8 mg | Stage 1- Venglustat 15 mg | Stage 2- Placebo | Stage 2- Venglustat 15 mg | Total Title | |
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Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months. | Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months. | Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months. | Participants from Stage 2 were randomized to receive 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months. | Participants from Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. | [Not Specified] | |
Overall Number of Baseline Participants | 78 | 78 | 80 | 123 | 119 | 478 | |
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Analysis was performed on all participants randomized in the study.
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Age, Continuous
Mean (Standard Deviation) Unit of measure: Years |
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Number Analyzed | 78 participants | 78 participants | 80 participants | 123 participants | 119 participants | 478 participants | |
42.6 (6.0) | 41.7 (6.9) | 43.6 (5.7) | 41.7 (6.9) | 42.1 (6.7) | 42.2 (6.5) | ||
Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 78 participants | 78 participants | 80 participants | 123 participants | 119 participants | 478 participants | |
Female |
37 47.4%
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31 39.7%
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34 42.5%
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46 37.4%
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48 40.3%
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196 41.0%
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Male |
41 52.6%
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47 60.3%
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46 57.5%
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77 62.6%
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71 59.7%
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282 59.0%
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Race (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 78 participants | 78 participants | 80 participants | 123 participants | 119 participants | 478 participants | |
American Indian or Alaska Native |
0 0.0%
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0 0.0%
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0 0.0%
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1 0.8%
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0 0.0%
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1 0.2%
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Asian |
27 34.6%
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27 34.6%
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27 33.8%
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52 42.3%
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55 46.2%
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188 39.3%
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Native Hawaiian or Other Pacific Islander |
0 0.0%
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0 0.0%
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1 1.3%
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0 0.0%
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0 0.0%
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1 0.2%
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Black or African American |
0 0.0%
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1 1.3%
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0 0.0%
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2 1.6%
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0 0.0%
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3 0.6%
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White |
48 61.5%
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50 64.1%
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52 65.0%
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66 53.7%
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64 53.8%
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280 58.6%
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More than one race |
1 1.3%
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0 0.0%
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0 0.0%
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0 0.0%
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0 0.0%
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1 0.2%
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Unknown or Not Reported |
2 2.6%
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0 0.0%
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0 0.0%
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2 1.6%
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0 0.0%
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4 0.8%
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Outcome Measures
Adverse Events
Limitations and Caveats
Following decision to discontinue EFC15392 based on the futility analysis, the two-steps analysis initially planned were not applicable, therefore only the final analysis of all safety endpoints (laboratory, vital sign, ECG, physical examination, BDI-II, and lens opacity) were performed on extended combined Stage 1 and Stage 2 safety population only.
More Information
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts
the PI's rights to discuss or publish trial results after the trial is completed.
The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
Results Point of Contact
Name/Title: | Trial Transparency Team |
Organization: | Sanofi aventis recherche & développement |
Phone: | 800-633-1610 ext 6# |
EMail: | Contact-US@sanofi.com |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Sanofi ( Genzyme, a Sanofi Company ) |
ClinicalTrials.gov Identifier: | NCT03523728 |
Other Study ID Numbers: |
EFC15392 2017-004084-12 ( EudraCT Number ) U1111-1202-0775 ( Other Identifier: UTN ) |
First Submitted: | May 1, 2018 |
First Posted: | May 14, 2018 |
Results First Submitted: | July 29, 2022 |
Results First Posted: | November 9, 2022 |
Last Update Posted: | November 9, 2022 |